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enrolling by invitation

An Extension Study to Evaluate Casimersen or Golodirsen in Patients With Duchenne Muscular Dystrophy

key information

study id #: NCT03532542

condition: Duchenne Muscular Dystrophy

status: enrolling by invitation


The main objective of this study is to evaluate the safety and tolerability of long-term treatment with casimersen or golodirsen in patients with Duchenne muscular dystrophy (DMD).

intervention: Casimersen, Golodirsen

mechanism of action: Exon-skipping to promote dystrophin production

results: https://clinicaltrials.gov/ct2/show/results/NCT03532542

last updated: July 19, 2019

study details

start date: August 2, 2018

estimated completion: June 30, 2026

phase of development: Phase 3

size / enrollment: 260

primary outcomes:

  • Number of patients with serious adverse events (SAEs) [Time Frame: Up to 30 days after the last infusion of study drug (assessed up to 148 weeks)]]

inclusion criteria:
• Completed a clinical trial evaluating casimersen or golodirsen, per protocol.
• Is between 7 and 23 years of age, inclusive, at enrollment.

study contacts

sponsor: Sarepta Therapeutics

investigators: Medical Director; Sarepta Therapeutics, Inc.

locations: United States, Belgium, Italy, Spain, United Kingdom